Success Story: Treating Pediatric Cancer and Blood Diseases at an Academic Medical Center

BY MARK SHWARTZ

WINTER 2001 - Michael Link, M.D. has been treating children with cancer for nearly three decades. As chief of the Division of Pediatric Hematology and Oncology at Packard, Link is all too familiar with the National Cancer Institute's (NCI) statistics on young cancer patients.

"According to the NCI, cancer is the leading cause of death by disease in children ages 1 to 14," he says. "One in 300 kids will be diagnosed with cancer by age 20."

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Despite these sobering numbers, dramatic advances have been made in the treatment of childhood cancers and blood diseases -- and Packard Hospital has been at the forefront of that success.

"What makes Packard a leader is that we have a superb, interdisciplinary clinical program to give comprehensive care to kids, as well as an outstanding academic staff affiliated with Stanford Medical School," says Harvey Cohen, M.D., Ph.D., Packard's chief of staff.

According to Cohen, Packard provides a remarkable breadth of expertise in a wide variety of areas, including radiation oncology and the treatment of brain tumors, neuroblastoma, hemophilia, leukemia, and Hodgkin disease. "Here, children get the latest treatments and the best supportive care for themselves and their families," he notes.

Center of Excellence

To emphasize Packard's commitment to pediatric hematology and oncology, Cohen points to the creation of the Center for Cancer and Blood Diseases. The Center is one of six Centers of Excellence designated as part of the Campaign for Lucile Packard Children's Hospital -- a half-billiondollar fundraising effort led by the Lucile Packard Foundation for Children's Health to shape preeminent programs at Packard in areas essential to children's health.

The Center will bring together an interdisciplinary team of physicians, nurses, and health care professionals to address the problems associated with diagnosing and treating childhood cancers and blood disorders, providing each patient with stateof- the-art medical technology in a child and family-friendly environment.

To encourage the discovery of new therapies and recruit world leaders in cancer research, the Campaign will establish new professorships in Stanford's Department of Pediatrics in the fields of cancer biology, bone marrow transplantation, and related areas.

The goal is monumental: to make Packard's Hematology and Oncology Division second to none.

Survival Rate

Looking back, there's been tremendous progress," says Philip Pizzo, M.D., dean of Stanford University School of Medicine and a nationally recognized authority on pediatric oncology.

"In 1960, about 25 percent of children diagnosed with cancer survived," Pizzo observes. "Today, close to 80 percent are long-term survivors. That's extraordinary, but it's not good enough. There are still too many children who do not respond to treatment."

According to the NCI, some 2,000 children die of cancer in the United States in each year -- the majority of deaths caused by high-risk leukemias or invasive malignancies of the central nervous system, primarily the brain. Pediatric specialists maintain that, with an infusion of dollars to support new research, clinical trials, and cutting-edge therapies, the overall mortality rate for pediatric cancers can be reduced to below 10 percent.

Cohen cites recent studies showing that young patients treated at university-affiliated pediatric cancer centers have a nearly 40 percent higher survival rate than those treated at community hospitals.

"It's best to have a close proximity to a university medical center with colleagues in adult medicine and the basic biosciences," Cohen notes. "You want to be where the experts are, because, when it comes to cancer, your first chance is your best chance."

The American Academy of Pediatrics agrees, recommending that all children and adolescents with newly diagnosed or recurrent malignancies be treated at a pediatric cancer center, like Packard, where each child has access to a team of oncology specialists.

When it comes to campus-wide research opportunities, says Dean Pizzo, Packard has barely scratched the surface. "By integrating further with Stanford Medical Center, the adult hospital, and the university at large, pediatric hematology and oncology at Packard will be enhanced," Pizzo maintains.

Pediatric vs. Adult Cancer

One reason for the high survival rate among young cancer patients today can be traced back to the late 1940s, when pediatric oncologists first recognized sharp differences between adult and childhood cancers.

In adults, cancer usually occurs when normal cells begin to grow abnormally. Adult cancers also tend to be localized, affecting only parts of organs, such as the lung, liver, breast, or prostate.

In children, cancers usually form in rapidly growing, developmental cell types in the blood, brain, soft tissue, and bone. Rather than being localized, pediatric cancers often have begun to spread throughout the body at the time of diagnosis.

While adult cancers frequently result from environmental factors, such as smoking, diet, or other exposures, the causes of childhood cancers remains unknown.

"In many cases we can treat kids more agressively than adults," notes Link. "They're usually healthy. They're not old and frail. In fact, kids are very responsive to treatment. Drugs that are ineffective in adults are spectacularly effective in children."

Chemotherapy, radiation, blood marrow transplantation, and surgery have proven highly successful in stopping many childhood cancers, says Link -- a result he attributes in large part to clinical trials, a cornerstone of pediatric oncology.

"Most adult cancer patients are seen in community hospitals and rarely get into clinical trials," Link explains, "but because most children are treated in cancer centers, they can participate in clinical trials. The result has been better therapies and a better understanding of cancers at a cellular and genetic level."

Link points to Packard's affiliation with the Children's Oncology Group (COG) -- an NCIsupported organization of 238 pediatric cancer centers around the world. Currently, Packard has an average of 75 COG clinical trials available to eligible cancer patients.

"The treatment of a child is more complex than that of an adult. It has to be designed so that it is specific to the disease, age, and development of the child," explains Cohen. "A child is a growing organism, and radiation can retard growth and affect intelligence.''

Link notes that on average, curing prostate cancer adds nine years to a man's life, but curing childhood cancer may add 69 years of life. "You want these to be high-quality years, so we have to be very cognizant of what we are doing to these kids by using less radiation and doing our utmost to preserve diseased organs and limbs," he says.

Future

In addition to offering such outstanding services as radiation oncology and bone marrow transplantation, Packard also is leading the development of new medicines for children. Gene therapy and recombinant DNA treatments being developed at Packard are providing hope for children with nonmalignant blood disorders such as aplastic anemia, sickle cell disease, and hemophilia.

"We have a phenomenal advantage, since many of the leading researchers in biomedicine and bioinformatics are right here at Stanford," observes Link.

While the outlook for children diagnosed with cancer and blood disorders has steadily improved, some illnesses -- such as brain tumors and neuroblastoma -- still have relatively low survival rates. "Pediatric cancers are fairly rare, so they are not on people's radar screens," Link notes. "But in 2010, one out every 450 young adults will be a survivor of childhood cancer, so clearly more work needs to be done."

Statistics are only part of the story. It's the personal side of pediatric oncology that gives Link his most satisfaction.

"We develop lifelong relationships with many families," he says. "I see patients that I treated coming back to the clinic with their own kids in tow. That's the lovely part of this whole thing -- taking someone through their darkest hour, then watching them grow up and lead a normal life."